Summary
Recombinant factor VIIa (rFVIIa; Novoseven™) is used for treatment of hemophilia patients
with inhibitors. There are poorly defined differences in clinical responsiveness between
individuals. Prior to licensure in the United States, rFVIIa was available through
the compassionate use program, during which two patients described in this study demonstrated
an excellent response. More recently, one of these individuals showed a sub-optimal
response to rFVIIa. One possible explanation for different treatment outcomes was
sequential therapy with prothrombin complex concentrates (PCC) followed by rFVIIa
in the compassionate use program. In support of this, an in vitro test showed that this patient had an exceptionally strong response to rFVIIa when
it was added to whole blood after the patient received PCC therapy. Results with other
patients supported this hypothesis. With further evaluation, a therapeutic approach
combining sequential PCC and rFVIIa may prove useful for treatment of bleeding refractory
to either agent used alone.
Keywords
Factor VIIa - prothrombin complex concentrates - haemophilia